VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy Received Positive Opinion by the (EMA) 


Pfizer issued a statement this week through its Media Relations that its drug VYNDAQEL® for treatment of Transthyretin Amyloid Cardiomyopathy received positive opinion by the  European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

About Vyndaqel® (tafamidis)

Vyndaqel is a once a day oral (61mg) medication that stabilizes the transthyretin protein and slows amyloid formation which is the cause of ATTR-CM. The EU has thus far not approved the marketing of Vyndaqel.


ATTR-CM is caused by deposits of misfolded amyloid protein that accumulate in the heart. Physicians are usually able to diagnose the disease only after the symptoms have become severe. From the time of diagnosis, patients may survive for less than four years.

Wild-type and hereditary ATTR-CM are sub-types of the disorder. The hereditary sub-type is caused by an abnormality in the transthyretin gene and the more common wild type is associated with aging. The former sub-type has been known to occur as early as age fifty while the latter generally appears in men over the age of sixty.

The ATTR-ACT Study

Pfizer’s application to the EMA was a result of the Phase 3 ATTR-ACT study of Vyndaqel. The positive results of the trial showed a reduction in mortality and in the frequency of hospitalizations due to cardiovascular occurrences. These patients were compared to patients with similar hereditary or wild-type ATTR-CM who received a placebo over a period of thirty months.

In various studies of Vyndaqel that was administered to ATTR-CM patients, the adverse events were similar to those experienced by patients receiving a placebo.

A final decision by the EC is expected within the next few months after its review of the CHMP’s opinion. If Vyndaqel is approved by the EC, it will be the first pharmacologic therapy in the European Union for the disorder.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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